Clinical characteristics and therapeutic outcomes of paroxysmal nocturnal hemoglobinuria patients in Turkey: a multicenter experience

Ann Hematol. 2021 Jul;100(7):1667-1675. doi: 10.1007/s00277-021-04554-4. Epub 2021 May 14.

Abstract

The aim of this study is to collect paroxysmal nocturnal hemoglobinuria (PNH) patient data from hematology centers all over Turkey in order to identify clinical features and management of PNH patients. Patients with PNH were evaluated by a retrospective review of medical records from 19 different institutions around Turkey. Patient demographics, medical history, laboratory findings, and PNH-specific information, including symptoms at the diagnosis, complications, erythrocyte, and granulocyte clone size, treatment, and causes of death were recorded. Sixty patients (28 males, 32 females) were identified. The median age was 33 (range; 17-77) years. Forty-six patients were diagnosed as classic PNH and 14 as secondary PNH. Fatigue and abdominal pain were the most frequent presenting symptoms. After eculizumab became available in Turkey, most of the patients (n = 31/46, 67.4%) were switched to eculizumab. Three patients with classic PNH underwent stem cell transplantation. The median survival time was 42 (range; 7-183 months) months. This study is the first and most comprehensive review of PNH cases in Turkey. It provided us useful information to find out the differences between our patients and literature, which may help us understand the disease.

Keywords: Eculizumab; Hemolysis; Paroxysmal nocturnal hemoglobinuria; Thrombosis.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Allografts
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Bone Marrow Diseases / complications
  • Drug Substitution
  • Female
  • Hemoglobinuria, Paroxysmal / drug therapy
  • Hemoglobinuria, Paroxysmal / epidemiology*
  • Hemoglobinuria, Paroxysmal / etiology
  • Hemoglobinuria, Paroxysmal / therapy
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Male
  • Middle Aged
  • Risk Factors
  • Survival Analysis
  • Symptom Assessment
  • Thrombophilia / etiology
  • Treatment Outcome
  • Turkey / epidemiology
  • Young Adult

Substances

  • Antibodies, Monoclonal, Humanized
  • Immunosuppressive Agents
  • eculizumab